PL2212424T3 - Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aav - Google Patents

Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aav

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Publication number
PL2212424T3
PL2212424T3 PL08836776T PL08836776T PL2212424T3 PL 2212424 T3 PL2212424 T3 PL 2212424T3 PL 08836776 T PL08836776 T PL 08836776T PL 08836776 T PL08836776 T PL 08836776T PL 2212424 T3 PL2212424 T3 PL 2212424T3
Authority
PL
Poland
Prior art keywords
gene delivery
motor neurons
aav vectors
peripheral injection
extensive gene
Prior art date
Application number
PL08836776T
Other languages
English (en)
Polish (pl)
Inventor
Martine Barkats
Original Assignee
Genethon
Centre Nat Rech Scient
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=39186802&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PL2212424(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genethon, Centre Nat Rech Scient filed Critical Genethon
Publication of PL2212424T3 publication Critical patent/PL2212424T3/pl

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P35/00Antineoplastic agents
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    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Pain & Pain Management (AREA)
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  • Anesthesiology (AREA)
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  • Rheumatology (AREA)
  • Psychiatry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Dermatology (AREA)
PL08836776T 2007-10-05 2008-10-03 Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aav PL2212424T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP07301435A EP2058401A1 (de) 2007-10-05 2007-10-05 Weitverbreitete Genverabreichung an Motorneuronen unter Verwendung einer peripheren Injektion von AAV-Vektoren
EP08836776.8A EP2212424B2 (de) 2007-10-05 2008-10-03 Weitreichende genverabreichung an motorneuronen unter verwendung einer peripheren injektion von aav-vektoren
PCT/EP2008/063297 WO2009043936A1 (en) 2007-10-05 2008-10-03 Widespread gene delivery to motor neurons using peripheral injection of aav vectors

Publications (1)

Publication Number Publication Date
PL2212424T3 true PL2212424T3 (pl) 2014-11-28

Family

ID=39186802

Family Applications (2)

Application Number Title Priority Date Filing Date
PL08836776T PL2212424T3 (pl) 2007-10-05 2008-10-03 Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aav
PL19158152.9T PL3505635T3 (pl) 2007-10-05 2008-10-03 Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aav

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL19158152.9T PL3505635T3 (pl) 2007-10-05 2008-10-03 Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aav

Country Status (19)

Country Link
US (3) US9926574B2 (de)
EP (5) EP2058401A1 (de)
JP (2) JP5620819B2 (de)
CN (2) CN101883859A (de)
CA (2) CA2701561C (de)
CY (2) CY1125608T1 (de)
DK (2) DK2212424T3 (de)
ES (3) ES2727034T3 (de)
FI (1) FI2212424T4 (de)
FR (2) FR20C1057I2 (de)
HR (2) HRP20220992T3 (de)
HU (2) HUE059864T2 (de)
LT (2) LT3505635T (de)
LU (1) LUC00285I2 (de)
NO (1) NO2022048I1 (de)
PL (2) PL2212424T3 (de)
PT (3) PT2212424E (de)
SI (2) SI3505635T1 (de)
WO (1) WO2009043936A1 (de)

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